期刊
ACS CHEMICAL BIOLOGY
卷 10, 期 4, 页码 914-924出版社
AMER CHEMICAL SOC
DOI: 10.1021/cb500697f
关键词
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资金
- Ministry of Education, Culture, Sports, Science, and Technology (MEXT) of Japan [21249013, 24591920]
- Research Program of Innovative Cell Biology from the Ministry of Education, Culture, Sports, Science, and Technology (MEXT) of Japan [231006]
- Platform for Drug Discovery from the Ministry of Education, Culture, Sports, Science, and Technology (MEXT) of Japan
- National Institute of Biomedical Innovation (NIBIO) [241011]
- CREST of Japan Science and Technology Agency (JST) [231038]
- Grants-in-Aid for Scientific Research [21249013, 24591920, 15K10050] Funding Source: KAKEN
Alternative splicing is a critical step where a limited number of human genes generate a complex and diverse proteome. Various diseases, including inherited diseases with abnormalities in the genome code, have been found to result in an aberrant mis-spliced transcript code with correlation to the resulting phenotype. Chemical compound-based and nucleic acid-based strategies are trying to target this mis-spliced transcript code. We will briefly mention about how to obtain splicing-modifying-compounds by high-throughput screening and overview of what is known about compounds that modify splicing pathways. The main focus will be on RNA-binding protein kinase inhibitors. In the main text, we will refer to diseases where splicing-modifying-compounds have been intensively investigated, with comparison to nucleic acid-based strategies. The information on their involvement in mis-splicing as well as nonsplicing events will be helpful in finding better compounds with less off-target effects for future implications in mis-splicing therapy.
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